Cytokines are secreted or membrane-presented molecules that mediate broad cellular
functions, including development, differentiation, growth and survival. Accordingly, the …

Sepsis is a life-threatening organ dysfunction caused by a dysregulated host response to
infection. Sepsis can be caused by a broad range of pathogens; however, bacterial …

CRISPR-Cas9 is a promising technology for genome editing. Here we use Cas9 nuclease-
induced double-strand break DNA (DSB) at the UROS locus to model and correct congenital …

Following periods of haematopoietic cell stress, such as after chemotherapy, radiotherapy,
infection and transplantation, patient outcomes are linked to the degree of immune …

The common cytokine receptor γ chain, γ c, is a component of the receptors for interleukin-2
(IL-2), IL-4, IL-7, IL-9, IL-15, and IL-21. Mutation of the gene encoding γ c results in X-linked …

The recombination-activating gene 1 (RAG1) and RAG2 proteins initiate the V (D) J
recombination process, which ultimately enables the generation of T cells and B cells with a …

Newborn screening for severe combined immunodeficiency, the most profound form of
primary immune system defects, has long been recognized as a measure that would …

Inborn errors of immunity (IEI) are diseases caused by genetic mutations that affect the
immune system's ability to fight pathogens, cope with the microbiota or regulate …

Ikaros/IKZF1 is an essential transcription factor expressed throughout hematopoiesis. IKZF1
is implicated in lymphocyte and myeloid differentiation and negative regulation of cell …

Ex vivo retrovirally mediated gene therapy has been shown within the last 20 yr to correct
the T cell immunodeficiency caused by γc-deficiency (SCID X1) and adenosine deaminase …